Takeda buys coeliac drug licence from COUR Pharmaceuticals

Takeda has bought a licence for a coeliac disease drug from COUR Pharmaceuticals, after the US biotech announced supportive phase 2a trial data.

Gastrointestinal diseases are one of the Japanese firm’s areas of focus and the company has bought an exclusive global licence for CNP-101/TAK-101, following the trial results in prevention of gluten induced immune activation in patients with coeliac disease.

COUR is set to receive up to $420 million in future payments and royalties on sales of any marketed products resulting from the licence.

TAK-101 is a potential first-in-class treatment targeting the aberrant immune response in coeliac disease, a serious condition where the ingestion of gluten leads to inflammation and damage in the small intestine.

The phase 2a results announced at a conference in Barcelona, Spain were from a randomised, double-blind placebo-controlled trial assessing the markers of potential efficacy and safety of the investigational medicine in 34 adults with proven coeliac disease.

At inclusion, patients had well-controlled biopsy proven coeliac disease, and after inclusion they underwent an oral gluten challenge.

Treatments were administered intravenously on day one and day eight. The gluten challenge began seven days after the second treatment administration and included 12 grams of gluten per day for three days followed by six grams of gluten per day for 11 days.

The primary endpoint was change from baseline in interferon-gamma (IFN-γ) spot forming units (SFUs) at day 6 after gluten challenge using a gliadin-specific enzyme-linked immunospot (ELISpot) assay.

This test is a direct measure of gluten-specific systemic T-cell activation in coeliac disease, and blocking this response suggests individuals with coeliac disease could be protected from the effects of gluten exposure.

The trial met its endpoint when 28 of the patients completed the 14-day gluten challenge protocol, with a mean change from baseline in measures of cell activation, although six discontinued due to gluten related symptoms.

Also seen was a trend in protection from small intestinal mucosal damage with deterioration. The most frequent adverse events in patients receiving TAK-101 that exceeded the frequency seen in placebo treated patients were nausea, headache, abdominal pain, and back pain.

No patient had clinically significant changes in vital signs, routine clinical labs, or serum cytokines/chemokines, gliadin-specific T-cell proliferation and cytokine secretion.

Asit Parikh, head of Gastroenterology Therapeutic Area Unit at Takeda, said: “Our collaboration with COUR has shown, for the first time, that it is possible to induce specific immune tolerance to a foreign antigen in autoimmune diseases such as coeliac disease.

“With our expertise in inflammatory diseases, Takeda is well positioned to further develop TAK-101 in pursuit of providing the first approved treatment option for patients with coeliac disease.”